I'm a Professor of Biomedical Engineering, Computer Science, and Biostatistics in the Institute of Genetic Medicine at Johns Hopkins University's School of Medicine. From 2005-2011 I was the Horvitz Professor of Computer Science and Director of the Center for Bioinformatics and Computational Biology at the University of Maryland, College Park. Before joining UMD, I was at The Institute for Genomic Research, where I sequenced the genomes of many bacteria, including those used in the 2001 anthrax attacks. At TIGR I was part of the Human Genome Project and the co-founder of the influenza virus sequencing project (which is when I first learned of the anti-vaccine movement). My research group develops software for DNA sequence analysis, and our (free) software is used by scientific laboratories around the globe. I did my B.A. and M.S. at Yale University, and my Ph.D. at Harvard University, and I have published over 200 scientific papers. Follow me on Facebook or Twitter (@stevensalzberg1), or just subscribe to my alternate blog, http://genome.fieldofscience.com.

Stem Cells Show Promise For Repairing Damaged Hearts

There was some very good news from the world of medicine just a couple of weeks ago. For the first time, stem cells were injected into the hearts of humans who had suffered serious heart damage, and patients improved dramatically. It appears that, as everyone hoped, the stem cells grew into new heart cells to replaced the damaged tissue. This is the promise of all stem cell research: to repair or replace damaged organs that otherwise would never recover. In principle, we can someday use the same technique to replace damaged livers, kidneys, spinal cords, cartilege, and virtually all other tissues in the human body.

In the new study, just published in The Lancet, a group of researchers led by Robert Bolli grew stem cells from patients’ own hearts, after the patients had suffered serious heart attacks, leaving their hearts permanently damaged. Bolli explained to CNN reporter Caleb Hellerman:

“Once you reach this stage of heart disease, you don’t get better. You can go down slowly, or go down quickly, but you’re going to go down.”

And yet, in this study, they did not “go down.” Instead, they got better. Bolli and colleagues collected a small amount of tissue from each patient’s own heart, and purified stem cells from that tissue. By using the patient’s own cells, there is no danger of rejection as there would be with cells from an unrelated donor.

They measured the patients’ heart function by how much blood was being pumpled through the left ventricle. The patients had an average Left Ventricular Ejection Fraction (LVEF) of 30.3% at the beginning of the study, an indication of very severe heart disease. Four months later, the 16 patients who received the stem cells had an average LVEF of 38.5%, while patients in the control group (who didn’t get the stem cells) showed no change. Even more dramatically, after one year the patients LVEF had improved further, to 42.5%.

Thus, remarkably, the cardiac stem cells seem to have “taken” in these patients, growing back into healthy cardiac cells in these severely ill patients. The researchers used MRI to measure the damaged heart tissue in 7 of their patients, and found that it had actually decreased by 30% after one year. In a companion trial at Cedars-Sinai Heart Institute in Los Angeles, Dr. Eduardo Marbán reported similarly positive results. Marbán told CNN’s Hellerman that the patients grew approximately 600 million new heart cells after the procedure, comparable to the number of cells that die in a serious heart attack.

One reason these findings are especially dramatic is that they show convincingly that the human heart contains stem cells that can re-grow into new heart cells. It is entirely possible that heart damage that has always been thought to be irreversible can be completely repaired – someday.

The results are very preliminary, and only a few patients have been treated so far, but this is a major triumph for stem cell research. The research in question used adult stem cells, but embryonic stem cells may prove even more effective, and may be easier to obtain because they don’t have to come directly from someone’s heart.* Heart disease is the leading cause of death in the U.S., and we should be pursuing every avenue that offers hope for better treatments, especially this very promising new direction. Those who oppose stem cell research – including embryonic stem cell research – should wake up and take notice: many lives are at stake.

*Disclaimer: Until June 2011, I was a member of the Maryland Stem Cell Research Commission, a state commission established by the legislature and the governor to promote human stem cell research through state-funded grants. The views expressed here, as always, are my own, and do not represent the Commission.

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Wouldn’t use of embryonic stem cells lead back ot the problem of rejection? It seems to me that if embryonic stem cells were used and the body started to reject and attack them there could be a worse problem. Rather than tying this to a fight over embryonic stem cells that many people have ethical and moral issues with, why not tie it into a goal for people to save their placentas (also a good source of stem cells, and not one that many people have issues with)?

The only problem with this story is the acceptance of the convention that it will take years for this therapy to reach anyone outside clinical trials. The response rate to this therapy was very close to 100 percent, meaning everyone who got the therapy got better. Based on more detailed reports, the people who had only recently gotten the therapy and therefore weren’t included in the first set of patients evaluated, have also responded. There are no significant side effects or risks so far. So why are we following the conventional path of moving to Phase II trials, then Phase III, which will take years and cost many, many millions of doallrs? Millions of people suffer from damaged hearts, and as Dr. Bolli notes, they decline, every time, and eventually die. This therapy works, and it exists right now. That it will be available “someday” is not acceptable. It is time for researchers and the FDA to develop a much greater sense of urgency regarding obvious breakthrough treatments, and to move much, much faster to deliver medical breakthroughs to people whose disease presents a much greater risk than a new therapy like the one described in this article. If this therapy is not moving into widespread use within a year, we should consider it a massive failure of our medical research and regulatory system. A 100 percent dramatic response rate in a small population with no side effects is not going to convert into a much worse outcome simply by giving it to more people in big randomized trials. We do need to know if there are risks that will energe in some small number of people, but we don’t need randomization to a control group to find that out, which emans this theraoy can start being delivered right now, along with learning more about it – from the people getting it. Time to deliver, folks. And while we are doing that, we should be asking, why are journalists so ready to accept an obviously wrong convention in this case – that it should take until “someday” for a medical breathrough to start saving lives? Do you think those countless people with damaged hearts and declining health, facing their own funerals, would agree? Whose risk is it?

I agree. The sooner, the better. They throw untried drugs that kill and damage out there immediately. But, hey, Big Pharma doesn’t want us to get well, just get drugged out as their pockets grow bigger. Let’s move this one to the front of the line because it will save a lot of people and money and care sooner, which is always better!

Gleevec treatment for CML is a precedent for speedy availability. During the Phase I trials for Gleevec, EVERYONE went into remission. Everyone. Considering Phase I trials are to establish dosing limits and there is very little expectation of efficacy, this was astonishing. Gleevec very quickly became available in clinical practice, ahead of phase II and phase III trials.

It was so fast that docs didn’t have guidelines for correct dosage. They couldn’t give patients an idea whether the remissions would be durable. But as you say, there were lives at stake.

Prior to Gleevec, CML was basically a death sentence, the only hope through very risky bone marrow transplants. Now it can be more like a manageable disease. And there now are second- and third-generation versions to address relapse on Gleevec.

This treatment appears to present a statistical equivalent. If it is not brought into practice with all due speed, I would be interested in knowing exactly why. I would wonder what entity had a financial stake in any delay.

“Immediate, broad use” can be interpreted to mean “available ASAP to anyone with a life-threatening heart condition that this treatment might possibly ameliorate”. Why should this option be denied to someone who is otherwise probably going to die? Two dozen patients is not insignificant.

However, creating the infrastructure to make this treatment widely available would not happen overnight.

Really Matthew? Faced with a terminal heart condition, you would refuse the treatment because it only saved 100% two dozen patients and not 100% of 2,000 patients?? Get real. Your’s, is the exact logic, swalker581 points out as the lunacy of of the FDA and some researchers that would rather watch one SURELYdie from their ailment, rather than provide them with an experimental treatment that COULD kill them. Please tell me you see the inverted logic in your reasoning!

“Those who oppose stem cell research – including embryonic stem cell research – should wake up and take notice…”

Maybe I haven’t been paying attention, but is there a movement that opposes ALL stem cell research? The controversy borne of the Clinton era ban on federal funding specifically involves embryonic stem cells. I don’t recall much opposition over the years to the type of research described in this article. I’m not sure what Dr. Salzberg is driving at with what appears to be an off topic comment.